Sickle Cell DiseaseNCT04523376
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
A study testing CliniMACS in people with Sickle Cell Disease and Thalassemia Major, run by Timothy Olson at 1 site in United States.
Trial summary
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta… Read more about this study
- Who is it for?
- Ages 2 to 25 years
- Conditions the study is for
- Sickle Cell Disease, Thalassemia Major
- What is being tested?
- CliniMACS
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 14 May 2020to 1 Oct 2025
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Severe Sickle Cell Disease
- Genotype: Hemoglobin SS, Hemoglobin SC, Hemoglobin SD, SOArab, or Hemoglobin SBeta thalassemia
- Must have at least one of the following disease manifestations
- Clinically symptomatic neurologic event (stroke) or any neurologic deficit lasting greater than 24 hours at any time prior to enrollment
- History of two or more episodes of vaso-occlusive events (VOE) per year in the 2 years preceding enrolment. Patients must be refractory to hydroxyurea, defined as developing VOE despite receiving hydroxyurea for at least 6 months. Patients who are intolerant of hydroxyurea may also be enrolled.
- Vaso-occlusive events include:
Show all 14Show fewer
- Acute chest syndrome
- Pain episodes requiring intravenous pain management and/or hospitalization
- Priapism
- Splenic sequestration (defined as a 2 g/dL drop in hemoglobin in the setting of an acutely enlarging spleen. This will be determined as part of clinical care and prior to the research)
- Administration of regular red blood cell (RBC) transfusion therapy, defined as receiving ≥ 8 RBC transfusions in the year preceding enrollment to prevent sickle cell-related complications of any kind per treating hematologist's judgment.
- Beta Thalassemia Major
- Genotype: Confirmed Beta Thalassemia genotype by molecular genetic testing (May include E/Beta0 and Beta0/Beta+ genotypes)
- Must meet clinical diagnosis of transfusion-dependent thalassemia, defined as need for ≥ 8 RBC transfusions per year in the two years preceding study enrollment.
Who cannot join
- Patients who do not meet disease, organ or infectious criteria.
- Previous Hematopoietic stem cell transplant (HSCT)
- Patients with no suitable unrelated donor available. Patients with suitable fully matched related donor are also not eligible.
- Pregnant females. All females of childbearing potential must have negative pregnancy test.
- Participation in a clinical trial in which the patient receives an investigational drug must be discontinued prior to the time of initiation of transplant therapy. Specifically transplant chemotherapy should not begin until at least 3 half-lives after last use of the investigational drug.
- Severe RBC alloimmunization, defined as inability to receive packed RBC transfusion therapy due to anti-RBC antibodies. Patients with high titer anti-donor human leukocyte antigen (HLA) antibodies detected on screening may be enrolled if they are willing to undergo HLA antibody desensitization therapy.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: Closely Matched Unrelated Donor Peripheral Blood Stem Cell Transplantation With TCRαβ+ T Cell and B Cell Depletion For Patients With Sickle Cell Disease and Thalassemia Major
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).
Read the detailed description
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo TCRαβ+ T cell and CD19+ B cell depletion of URD grafts using the CliniMACS device in patients with SCD and BTM. Apart from CliniMACS-based cell processing, PSCT will be performed according to current standards of care in the Children's Hospital of Philadelphia (CHOP) Cell Therapy and Transplant Section, including the use of a standard chemotherapy conditioning regimen and standard follow-up laboratory assessments. The study will determine efficacy of this strategy in terms of engraftment, rates of acute and chronic Graft versus Host Disease (GvHD), and one-year overall and event-free survival.
- Conditions
- Sickle Cell Disease, Thalassemia Major
- Being tested
- CliniMACS
- Medical subject headings
- Anemia, Sickle Cell, beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CliniMACSDevice
Peripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique
| Study group | Role | What they receive |
|---|---|---|
| Sickle Cell Disease | Experimental | Patients with Sickle Cell Disease (SCD) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors. Receives: Device: CliniMACS |
| Beta Thalassemias Major | Experimental | Patients with Beta Thalassemias Major (BTM) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors. Receives: Device: CliniMACS |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Graft Failure
Number of patients with primary graft failure (defined as no evidence of neutrophil engraftment by day +30 after stem cell infusion) and secondary graft failure (defined as ANC <500 for at least 7-10 days after initial engraftment occurs in the absence of known infection or drug-mediated suppression, and confirmed by hypocellular bone marrow biopsy and/or total donor chimerism percentage from blood or bone marrow < 10 percent)
Measured: Up to 1 year post-transplantation
Time to Neutrophil Engraftment
Number of days to neutrophil engraftment (first day of ANC >500/µl for the first of 3 consecutive days)
Measured: Up to 60 days post-transplantation
Incidence of Acute Graft vs. Host Disease (GVHD)
Acute GvHD was assessed by the number of patients who developed acute graft-versus-host disease, graded according to current Center for International Bone Marrow Transplant Registry (CIBMTR) reporting guidelines. Grading follows established criteria based on the severity of skin, liver, and gastrointestinal involvement, including extent of rash, bilirubin elevation, and gastrointestinal symptoms (e.g., diarrhea volume). Evaluation was performed by clinical assessment and laboratory data consistent with standard transplant-related acute GvHD grading practices.
Measured: Up to 100 days post-transplantation
Incidence of Chronic Graft vs. Host Disease (GVHD)
Number of patients with Grade II-IV acute GVHD, Severe Grade III-IV acute GVHD, and Chronic Extensive GVHD
Measured: Up to two years post-transplantation
Secondary outcomes (4)
Number of Deaths Due to Treatment
Number of subjects deaths that were related to study treatment
Measured: Up to 100 days post-transplantation
Probability of Event-free Survival (EFS)
Number of patients without complications or events
Measured: Up to 1 year post-transplantation
Probability of Overall Survival (OS)
Number of patients with the following survival outcome: one-year overall survival (OS)
Measured: 1 year post-transplantation
Incidence of Viral Reactivation and Symptomatic Viral Infection
Number of patients experiencing viral reactivation requiring therapy and symptomatic viral infections, including CMV, adenovirus, and EBV
Measured: Up to 1 year post-transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2020
- Study start14 May 2020
- Primary completion23 Jun 2025
- Study completion1 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (63)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Timothy Olson
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Timothy Olson, Children's Hospital of Philadelphia
- Investigators
- Timothy Olson, MD, PhD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04523376
- Sponsor's protocol ID
- 19-017141
- Status last verified
- Mar 2026
- First submitted
- 19 Aug 2020
- Last updated
- 28 Apr 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- No
Publications
Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017 Apr 20;376(16):1561-1573. doi: 10.1056/NEJMra1510865. No abstract available.
PubMed 28423290Maitra P, Caughey M, Robinson L, Desai PC, Jones S, Nouraie M, Gladwin MT, Hinderliter A, Cai J, Ataga KI. Risk factors for mortality in adult patients with sickle cell disease: a meta-analysis of studies in North America and Europe. Haematologica. 2017 Apr;102(4):626-636. doi: 10.3324/haematol.2016.153791. Epub 2017 Jan 19.
PubMed 28104703Arnold SD, Jin Z, Sands S, Bhatia M, Kung AL, Satwani P. Allogeneic Hematopoietic Cell Transplantation for Children with Sickle Cell Disease Is Beneficial and Cost-Effective: A Single-Center Analysis. Biol Blood Marrow Transplant. 2015 Jul;21(7):1258-65. doi: 10.1016/j.bbmt.2015.01.010. Epub 2015 Jan 20.
PubMed 25615608Gardner K, Douiri A, Drasar E, Allman M, Mwirigi A, Awogbade M, Thein SL. Survival in adults with sickle cell disease in a high-income setting. Blood. 2016 Sep 8;128(10):1436-8. doi: 10.1182/blood-2016-05-716910. Epub 2016 Jul 20. No abstract available.
PubMed 27439910Nevitt SJ, Jones AP, Howard J. Hydroxyurea (hydroxycarbamide) for sickle cell disease. Cochrane Database Syst Rev. 2017 Apr 20;4(4):CD002202. doi: 10.1002/14651858.CD002202.pub2.
PubMed 28426137Torres L, Conran N. Emerging pharmacotherapeutic approaches for the management of sickle cell disease. Expert Opin Pharmacother. 2019 Feb;20(2):173-186. doi: 10.1080/14656566.2018.1548610. Epub 2018 Nov 30.
PubMed 30499731Chou ST, Fasano RM. Management of Patients with Sickle Cell Disease Using Transfusion Therapy: Guidelines and Complications. Hematol Oncol Clin North Am. 2016 Jun;30(3):591-608. doi: 10.1016/j.hoc.2016.01.011.
PubMed 27112998Sayani FA, Kwiatkowski JL. Increasing prevalence of thalassemia in America: Implications for primary care. Ann Med. 2015;47(7):592-604. doi: 10.3109/07853890.2015.1091942. Epub 2015 Nov 5.
PubMed 26541064Kwiatkowski JL. Current recommendations for chelation for transfusion-dependent thalassemia. Ann N Y Acad Sci. 2016 Mar;1368(1):107-14. doi: 10.1111/nyas.13088.
PubMed 27186943Porter J. Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):361-370. doi: 10.1182/asheducation-2018.1.361.
PubMed 30504333Gluckman E. Allogeneic transplantation strategies including haploidentical transplantation in sickle cell disease. Hematology Am Soc Hematol Educ Program. 2013;2013:370-6. doi: 10.1182/asheducation-2013.1.370.
PubMed 24319206Vermylen C, Cornu G. Hematopoietic stem cell transplantation for sickle cell anemia. Curr Opin Hematol. 1997 Nov;4(6):377-80. doi: 10.1097/00062752-199704060-00003.
PubMed 9358992Walters MC, Patience M, Leisenring W, Eckman JR, Scott JP, Mentzer WC, Davies SC, Ohene-Frempong K, Bernaudin F, Matthews DC, Storb R, Sullivan KM. Bone marrow transplantation for sickle cell disease. N Engl J Med. 1996 Aug 8;335(6):369-76. doi: 10.1056/NEJM199608083350601.
PubMed 8663884Bernaudin F, Socie G, Kuentz M, Chevret S, Duval M, Bertrand Y, Vannier JP, Yakouben K, Thuret I, Bordigoni P, Fischer A, Lutz P, Stephan JL, Dhedin N, Plouvier E, Margueritte G, Bories D, Verlhac S, Esperou H, Coic L, Vernant JP, Gluckman E; SFGM-TC. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. 2007 Oct 1;110(7):2749-56. doi: 10.1182/blood-2007-03-079665. Epub 2007 Jul 2.
PubMed 17606762Panepinto JA, Walters MC, Carreras J, Marsh J, Bredeson CN, Gale RP, Hale GA, Horan J, Hows JM, Klein JP, Pasquini R, Roberts I, Sullivan K, Eapen M, Ferster A; Non-Malignant Marrow Disorders Working Committee, Center for International Blood and Marrow Transplant Research. Matched-related donor transplantation for sickle cell disease: report from the Center for International Blood and Transplant Research. Br J Haematol. 2007 Jun;137(5):479-85. doi: 10.1111/j.1365-2141.2007.06592.x. Epub 2007 Apr 24.
PubMed 17459050
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Closely Matched Unrelated Donor Peripheral Blood Stem Cell Transplantation With TCRαβ+ T Cell and B Cell Depletion For Patients With Sickle Cell Disease and Thalassemia Major
- ClinicalTrials.gov ID
- NCT04523376
- Acronym / sponsor ID
- 19-017141
- Conditions
- Sickle Cell Disease, Thalassemia Major
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Timothy OlsonUniversity, hospital or other
- Study start
- 14 May 2020 (actual)
- Primary completion
- 23 Jun 2025 (actual)
- Study completion
- 1 Oct 2025 (actual)
- First posted
- 21 Aug 2020 (actual)
- Results
- Posted 28 Apr 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).
Detailed description
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo TCRαβ+ T cell and CD19+ B cell depletion of URD grafts using the CliniMACS device in patients with SCD and BTM. Apart from CliniMACS-based cell processing, PSCT will be performed according to current standards of care in the Children's Hospital of Philadelphia (CHOP) Cell Therapy and Transplant Section, including the use of a standard chemotherapy conditioning regimen and standard follow-up laboratory assessments. The study will determine efficacy of this strategy in terms of engraftment, rates of acute and chronic Graft versus Host Disease (GvHD), and one-year overall and event-free survival.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sickle Cell Disease | Experimental | Device: CliniMACSPatients with Sickle Cell Disease (SCD) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors. |
| Beta Thalassemias Major | Experimental | Device: CliniMACSPatients with Beta Thalassemias Major (BTM) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors. |
Who may qualify
- Ages
- 2 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- Severe Sickle Cell Disease
- Genotype: Hemoglobin SS, Hemoglobin SC, Hemoglobin SD, SOArab, or Hemoglobin SBeta thalassemia
- Must have at least one of the following disease manifestations
- Clinically symptomatic neurologic event (stroke) or any neurologic deficit lasting greater than 24 hours at any time prior to enrollment
- History of two or more episodes of vaso-occlusive events (VOE) per year in the 2 years preceding enrolment. Patients must be refractory to hydroxyurea, defined as developing VOE despite receiving hydroxyurea for at least 6 months. Patients who are intolerant of hydroxyurea may also be enrolled.
Key exclusion criteria (6 listed)
- Patients who do not meet disease, organ or infectious criteria.
- Previous Hematopoietic stem cell transplant (HSCT)
- Patients with no suitable unrelated donor available. Patients with suitable fully matched related donor are also not eligible.
- Pregnant females. All females of childbearing potential must have negative pregnancy test.
- Participation in a clinical trial in which the patient receives an investigational drug must be discontinued prior to the time of initiation of transplant therapy. Specifically transplant chemotherapy should not begin until at least 3 half-lives after last use of the investigational drug.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CliniMACSPeripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique | Device | Beta Thalassemias Major, Sickle Cell Disease |
Primary outcome
Rate of Graft Failure
Time frame: Up to 1 year post-transplantation
Number of patients with primary graft failure (defined as no evidence of neutrophil engraftment by day +30 after stem cell infusion) and secondary graft failure (defined as ANC <500 for at least 7-10 days after initial engraftment occurs in the absence of known infection or drug-mediated suppression, and confirmed by hypocellular bone marrow biopsy and/or total donor chimerism percentage from blood or bone marrow < 10 percent)
Time to Neutrophil Engraftment
Time frame: Up to 60 days post-transplantation
Number of days to neutrophil engraftment (first day of ANC >500/µl for the first of 3 consecutive days)
Incidence of Acute Graft vs. Host Disease (GVHD)
Time frame: Up to 100 days post-transplantation
Acute GvHD was assessed by the number of patients who developed acute graft-versus-host disease, graded according to current Center for International Bone Marrow Transplant Registry (CIBMTR) reporting guidelines. Grading follows established criteria based on the severity of skin, liver, and gastrointestinal involvement, including extent of rash, bilirubin elevation, and gastrointestinal symptoms (e.g., diarrhea volume). Evaluation was performed by clinical assessment and laboratory data consistent with standard transplant-related acute GvHD grading practices.
Incidence of Chronic Graft vs. Host Disease (GVHD)
Time frame: Up to two years post-transplantation
Number of patients with Grade II-IV acute GVHD, Severe Grade III-IV acute GVHD, and Chronic Extensive GVHD
4 secondary outcomes
Number of Deaths Due to Treatment
Time frame: Up to 100 days post-transplantation
Probability of Event-free Survival (EFS)
Time frame: Up to 1 year post-transplantation
Probability of Overall Survival (OS)
Time frame: 1 year post-transplantation
Incidence of Viral Reactivation and Symptomatic Viral Infection
Time frame: Up to 1 year post-transplantation
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 28423290Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017 Apr 20;376(16):1561-1573. doi: 10.1056/NEJMra1510865. No abstract available.
- Background: PubMed 28104703Maitra P, Caughey M, Robinson L, Desai PC, Jones S, Nouraie M, Gladwin MT, Hinderliter A, Cai J, Ataga KI. Risk factors for mortality in adult patients with sickle cell disease: a meta-analysis of studies in North America and Europe. Haematologica. 2017 Apr;102(4):626-636. doi: 10.3324/haematol.2016.153791. Epub 2017 Jan 19.
- Background: PubMed 25615608Arnold SD, Jin Z, Sands S, Bhatia M, Kung AL, Satwani P. Allogeneic Hematopoietic Cell Transplantation for Children with Sickle Cell Disease Is Beneficial and Cost-Effective: A Single-Center Analysis. Biol Blood Marrow Transplant. 2015 Jul;21(7):1258-65. doi: 10.1016/j.bbmt.2015.01.010. Epub 2015 Jan 20.
- Background: PubMed 27439910Gardner K, Douiri A, Drasar E, Allman M, Mwirigi A, Awogbade M, Thein SL. Survival in adults with sickle cell disease in a high-income setting. Blood. 2016 Sep 8;128(10):1436-8. doi: 10.1182/blood-2016-05-716910. Epub 2016 Jul 20. No abstract available.
- Background: PubMed 28426137Nevitt SJ, Jones AP, Howard J. Hydroxyurea (hydroxycarbamide) for sickle cell disease. Cochrane Database Syst Rev. 2017 Apr 20;4(4):CD002202. doi: 10.1002/14651858.CD002202.pub2.
- Background: PubMed 30499731Torres L, Conran N. Emerging pharmacotherapeutic approaches for the management of sickle cell disease. Expert Opin Pharmacother. 2019 Feb;20(2):173-186. doi: 10.1080/14656566.2018.1548610. Epub 2018 Nov 30.
- Background: PubMed 27112998Chou ST, Fasano RM. Management of Patients with Sickle Cell Disease Using Transfusion Therapy: Guidelines and Complications. Hematol Oncol Clin North Am. 2016 Jun;30(3):591-608. doi: 10.1016/j.hoc.2016.01.011.
- Background: PubMed 26541064Sayani FA, Kwiatkowski JL. Increasing prevalence of thalassemia in America: Implications for primary care. Ann Med. 2015;47(7):592-604. doi: 10.3109/07853890.2015.1091942. Epub 2015 Nov 5.
- Background: PubMed 27186943Kwiatkowski JL. Current recommendations for chelation for transfusion-dependent thalassemia. Ann N Y Acad Sci. 2016 Mar;1368(1):107-14. doi: 10.1111/nyas.13088.
- Background: PubMed 30504333Porter J. Beyond transfusion therapy: new therapies in thalassemia including drugs, alternate donor transplant, and gene therapy. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):361-370. doi: 10.1182/asheducation-2018.1.361.
- Background: PubMed 24319206Gluckman E. Allogeneic transplantation strategies including haploidentical transplantation in sickle cell disease. Hematology Am Soc Hematol Educ Program. 2013;2013:370-6. doi: 10.1182/asheducation-2013.1.370.
- Background: PubMed 9358992Vermylen C, Cornu G. Hematopoietic stem cell transplantation for sickle cell anemia. Curr Opin Hematol. 1997 Nov;4(6):377-80. doi: 10.1097/00062752-199704060-00003.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Timothy Olson, MD, PhD principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CliniMACS in people with Sickle Cell Disease and Thalassemia Major, run by Timothy Olson at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of Graft Failure (time frame: Up to 1 year post-transplantation). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04523376, which also lists 63 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2026.